even though the nonlysosomal glycogenolytic system is normal, glycogen still accumulates in the lysosomes in almost all tissues, but produces especially severe effects in skeletal and cardiac muscle leading to early mortality.
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... Synthase 1 Synthesis as an Approach for Substrate Reduction Therapy of Pompe Disease. Clayton NP, Nelson CA, Weeden T, Taylor KM, Moreland RJ, Scheule RK, Phillips L, Leger ...
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